Santa Cecilia

Researchers are evaluating the safety and effectiveness of GTX-102 in people with Angelman syndrome, a genetic condition, across different ages and genetic types. This Phase 2 open-label basket study includes subprotocols A, B, C, and D, each following a similar design with Screening, Loading, and Maintenance periods. Some subprotocols involve randomization, and participants can continue treatment in a long-term extension after completing the study. Participants receive GTX-102 via intrathecal injections every three months. Dosing starts at a lower level and increases until a target dose is reached. Different subprotocols include groups based on age and Angelman syndrome genotype, with some receiving no treatment initially before starting GTX-102. The study evaluates treatment effects in groups ranging from children as young as 1 year old to adults up to 65 years old. Throughout the study, participants undergo scheduled visits with assessments including cognitive and motor function tests, behavior rating scales, and safety monitoring for adverse events. Researchers collect data up to about 17 months from baseline, with monitoring of treatment-emergent and serious adverse events. Participants also have imaging and lab tests like lumbar punctures and blood work. After the initial study, participants may opt to continue in a long-term extension to further assess GTX-102s effects and safety.

Researchers are evaluating the effects of vicadrostat combined with empagliflozin in adults who have type 2 diabetes, high blood pressure, and cardiovascular disease but no history of heart failure. The study aims to assess whether this combination can help reduce cardiovascular risks compared to a placebo with empagliflozin. This Phase III trial involves adults with these conditions who are already receiving treatment for them. Participants are randomly assigned to one of two groups. One group takes vicadrostat and empagliflozin tablets daily, while the other group takes placebo tablets that look like vicadrostat but have no active medicine, alongside empagliflozin. Treatment lasts from two and a half years up to four years and three months. All participants continue their usual medications for diabetes, blood pressure, and heart disease during the study. Throughout the study, lasting up to four years and three months, participants visit the study site regularly for health checks and blood samples. Doctors monitor cardiovascular events and any side effects experienced. The main outcome measured is the time until the first cardiovascular death or heart failure event. Other health indicators like blood pressure and kidney function are also tracked to understand the effects of the treatment combination.

Researchers are evaluating the safety and effectiveness of ficerafusp alfa combined with pembrolizumab compared to placebo with pembrolizumab for adults with first-line PD-L1-positive, recurrent or metastatic Head and Neck Squamous Cell Carcinoma. This study focuses on the dual targeting of EGFR and TGF-beta, which contribute to tumor growth and spread. It includes both phase 2 and phase 3 parts to identify the best dose and to compare treatment outcomes. In phase 2, participants are randomized into three groups receiving either higher or lower doses of ficerafusp alfa plus pembrolizumab, or placebo plus pembrolizumab. In phase 3, participants receive the selected optimal biologic dose of ficerafusp alfa with pembrolizumab or placebo with pembrolizumab in a 21 ratio. Treatments are given weekly or every three weeks depending on the drug, with careful monitoring throughout the study. Participants will undergo tumor biopsies or provide archival tissue, and receive regular assessments including imaging scans and lab tests to measure tumor response and safety. Researchers will track side effects, response rates, survival, and quality of life using standardized criteria over approximately 1 to 3 years. Safety monitoring continues up to 90 days after treatment ends. The total study duration extends through long-term follow-up to evaluate overall outcomes.